Deals of the Week: Sanofi-Pasteur/CureVac, Shire/Shionogi, Genzyme/CFF, Medicis/Graceway
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Cystic Fibrosis Market Snapshot: Disease-Modifying Drugs Elusive 24 Years After Discovery Of Root Cause
The discovery in 1989 of the underlying genetics causing cystic fibrosis was expected to spur broad development of disease-modifying therapies. As of 2013, however, only one such drug is on the market, for a small subset of CF patients, and a few other candidates are in clinical development.
Agios brings in a $78 million in financing from both previous venture investors and new public investor funds, as it embarks on research of rare diseases caused by an inborn error of metabolism.
The pioneering U.S. biotech will look to partner its five human embryonic stem cell research programs while focusing entirely on two mid-stage cancer assets.